Cell and gene therapy (CGT) products and pipeline

In 2019, the US Food and Drug Administration (FDA) predicted approval by 2025 of 10 to 20 CGT products a year, based on an assessment of the current pipeline and clinical success rates of these products at that time. Experience has since taught us that the development of cell and gene therapies is perhaps more difficult than anticipated in 2019.

A number of clinical trials have seen temporary clinical holds or have been terminated. Some pipeline products have been shelved based on lack of financial viability. Nevertheless, the submission of FDA Investigational New Drug (IND) applications for cell and gene therapies has continued. Additionally, the FDA is furthering its application guidance, while other FDA communications suggest the agency is pursuing a broader scope of accepted trial outcomes to increase the number and speed of durable therapies reaching the market.